Experimental drug for brittle bones tested in small trial

NCT ID NCT05231668

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tested a single dose of a drug called SAR439459 in 16 adults with osteogenesis imperfecta (brittle bone disease). The drug aims to block a protein that may weaken bones. The study focused on safety and how the drug moves through the body, but it was terminated early, so results are limited.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

SAR439459 (a monoclonal antibody that blocks TGFβ)

What this could lead to

If successful, this could point toward a new treatment to strengthen bones and reduce fractures in people with osteogenesis imperfecta.

What could go wrong

This was a very early, small Phase 1 trial that was terminated, so results are limited. The drug may not prove effective or safe in larger studies.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

osteogenesis imperfecta

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Baylor College of Medicine - Site Number:8400003

    Houston, Texas, 77030, United States

  • Bone Research and Education Centre_Site Number :1240003

    Oakville, Ontario, L6M 1M1, Canada

  • Cincinnati Children's Hospital Medical Center Site Number : 8400010

    Cincinnati, Ohio, 45229, United States

  • Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002

    Clayton, Victoria, 3168, Australia

  • Hopital Edouard Herriot _Site Number :2500002

    Lyon, 69003, France

  • Hopital Lariboisiere_Site Number :2500001

    Paris, 75010, France

  • Indiana University School of Medicine_Site Number: 8400002

    Indianapolis, Indiana, 46202, United States

  • Kennedy Krieger Institute_Site number 8400004

    Baltimore, Maryland, 21205, United States

  • Toronto general Hospital_Site Number :1240002

    Toronto, M5G 2C4, Canada

  • UCLA Health_Site Number: 8400006

    Los Angeles, California, 90095, United States

  • Vanderbilt University Site Number : 8400011

    Nashville, Tennessee, 37232, United States

  • Westmead Hospital_Site Number :0360003

    Westmead, New South Wales, 2145, Australia

  • Yale University - Site Number:8400007

    New Haven, Connecticut, 06510, United States